New pill treats cystic fibrosis at its source

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Raleigh, N.C. — For many cystic fibrosis patients the only available option is to manage the symptoms, but a new drug aims to treat the disease at its source.

Cystic fibrosis affects the airways and the digestive tract by causing mucus to build up in those areas. The disease can be life-threatening.

20-year-old Patrick Fulkerson has been going to Duke to treat his cystic fibrosis his whole life, a treatment regimen which has involved lung function tests, hospitalizations and home treatments to clear the mucus from his airways.

"My earliest memory is seeing Duke for the first time," Fulkerson said.

After he started taking the new drug, Kalydeco, his lung function test results improved. Kalydeco is designed to treat a gene mutation that 4 to 5 percent of cystic fibrosis patients have.

"Kalydeco is a pill that's taken by mouth, and can actually change the mutant protein and make it function more normally," said Dr. Judith Voynow, director of the Duke Cystic Fibrosis Center. "We really could completely change the landscape for cystic fibrosis and hopefully prevent any manifestations of the disease."

The pill has been approved for patients with the same mutation who are over 6 years of age and is being studied in younger patients with promising results. Voynow said Kalydeco has opened the door for similar research targeting the 1,500 other gene mutations that cause cystic fibrosis.

Fulkerson said he only expected Kalydeco to be another method to control his cystic fibrosis.

"It's in conjunction with keeping up with my treatments, exercising and eating and drinking properly," he said.